Global Patent Index - EP 3020404 A1

EP 3020404 A1 20160518 - Caffeine for the treatment of myotonic dystrophy type 1 and type 2

Title (en)

Caffeine for the treatment of myotonic dystrophy type 1 and type 2

Title (de)

Koffein zur Behandlung von myotoner Dystrophie Typ 1 und Typ 2

Title (fr)

Caféine pour le traitement de la dystrophie myotonique de type 1 et de type 2

Publication

EP 3020404 A1 20160518 (EN)

Application

EP 14382450 A 20141114

Priority

EP 14382450 A 20141114

Abstract (en)

The present invention relates to caffeine for use in the treatment of myotonic dystrophy type 1 and type 2. The present invention also relates to compositions comprising caffeine for use in the treatment of myotonic dystrophy type 1 and type 2.

IPC 8 full level

A61K 31/522 (2006.01); A61P 21/00 (2006.01); A61P 43/00 (2006.01)

CPC (source: EP US)

A23C 9/152 (2013.01 - US); A23L 2/52 (2013.01 - EP US); A23L 7/10 (2016.07 - US); A23L 33/10 (2016.07 - US); A23L 33/105 (2016.07 - EP US); A23L 33/30 (2016.07 - US); A61K 31/522 (2013.01 - EP US); A61P 21/00 (2017.12 - EP); A61P 43/00 (2017.12 - EP); A23V 2002/00 (2013.01 - EP US)

C-Set (source: EP US)

  1. A61K 31/522 + A61K 2300/00
  2. A23V 2002/00 + A23V 2200/316 + A23V 2250/2108

Citation (applicant)

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  • WO 03020977 A2 20030313 - UNIV GLASGOW [GB], et al
  • SUOMINEN, T. ET AL.: "Population frequency of myotonic dystrophy: higher than expected frequency of myotonic dystrophy type 2 (DM2) mutation in Finland", EUR J HUM GENET, vol. 19, no. 7, 2011, pages 776 - 82
  • GOMES-PEREIRA, M. ET AL.: "Myotonic dystrophy mouse models: towards rational therapy development", TRENDS MOL MED., vol. 17, no. 9, 2011, pages 506 - 17
  • WARF, M.; NAKAMORI, M.; MATTHYS, C.; THORNTON, C.; BERGLUND, J.: "Pentamidine reverses the splicing defects associated with myotonic dystrophy", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 106, 2009, pages 18551 - 18556
  • C. THORNTON, C.; BERGLUND, J.: "Pentamidine reverses the splicing defects associated with myotonic dystrophy", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 106, 2009, pages 18551 - 18556
  • COONROD, L. ET AL.: "Reducing levels of toxic RNA with small molecules", ACS CHEM BIOL., vol. 8, no. 11, 2013, pages 2528 - 37
  • A., HALEY, M.; THORNTON, C. ET AL.: "Reducing levels of toxic RNA with small molecules", ACS CHEMICAL BIOLOGY, vol. 8, 2013, pages 2528 - 2537
  • PARKESH, R.; CHILDS-DISNEY, J.; NAKAMORI, M.; KUMAR, A.; WANG, E.; WANG, T.; HOSKINS, J.; TRAN, T.; HOUSMAN, D.; THORNTON, C. ET A: "Design of a Bioactive Small Molecule That Targets the Myotonic Dystrophy Type 1 RNA via an RNA Motif-Ligand Database and Chemical Similarity Searching", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 134, 2012, pages 4731 - 4742
  • GAREISS, P.; SOBCZAK, K.; MCNAUGHTON, B.; PALDE, P.; THORNTON, C.; MILLER, B.: "Dynamic combinatorial selection of molecules capable of inhibiting the (CUG) repeat RNA-MBNL1 interaction in vitro: discovery of lead compounds targeting myotonic dystrophy (DM1", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 130, 2008, pages 16254 - 16261
  • GARCIA-LOPEZ, A.; LLAMUSI, B.; ORZAEZ, M.; PEREZ-PAYA, E.; ARTERO, R.: "In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, vol. 108, 2011, pages 11866 - 11871
  • JAHROMI, A.H.; NGUYEN, L.; FU, Y.; MILLER, K.A.; BARANGER, A.M.; ZIMMERMAN, S.C.: "A novel CUG(exp). MBNL1 inhibitor with therapeutic potential for myotonic dystrophy type 1", ACS CHEMICAL BIOLOGY, vol. 8, 2013, pages 1037 - 1043
  • WONG, C.H.; NGUYEN, L.; PEH, J.; LUU, L.M.; SANCHEZ, J.S.; RICHARDSON, S.L.; TUCCINARDI, T.; TSOI, H.; CHAN, W.Y.; CHAN, H.Y. ET A: "Targeting Toxic RNAs that Cause Myotonic Dystrophy Type 1 (DM1) with a Bisamidinium Inhibitor", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 136, 2014, pages 6355 - 6361
  • CHILDS-DISNEY, J.; STEPNIAK-KONIECZNA, E.; TRAN, T.; YILDIRIM, I.; PARK, H.; CHEN, C.; HOSKINS, J.; SOUTHALL, N.; MARUGAN, J.; PAT: "Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules", NATURE COMMUNICATIONS, vol. 4, 2013, pages 2044
  • HOSKINS, J.W.; OFORI, L.O.; CHEN, C.Z.; KUMAR, A.; SOBCZAK, K.; NAKAMORI, M.; SOUTHALL, N.; PATNAIK, S.; MARUGAN, J.J.; ZHENG, W.: "Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects", NUCLEIC ACIDS RESEARCH, vol. 42, 2014, pages 6591 - 6602
  • GOMES-PEREIRA ET AL., NUC. ACIDS RESEARCH, vol. 32, no. 9, 2004, pages 2865 - 2872
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Citation (search report)

  • [XI] J CAMPBELL ET AL: "Research Article Open Access Review Article Open Access Congenital Myotonic Dystrophy", J NEUROL NEUROPHYSIOL, 23 January 2012 (2012-01-23), pages S7 - 001, XP055186364, ISSN: 2155-9562, DOI: 10.4172/2155-9562.S7-001
  • [XI] KELLER C ET AL: "Congenital myotonic dystrophy requiring prolonged endotracheal and noninvasive assisted ventilation: not a uniformly fatal condition", PEDIATRICS, AMERICAN ACADEMY OF PEDIATRICS, EVANSTON, IL, US, vol. 101, no. 4, 1 April 1998 (1998-04-01), pages 704 - 706, XP009184025, ISSN: 0031-4005
  • [Y] QUEUDEVILLE M ET AL: "Congenital myotonic dystrophy with diaphragmatic paresis ; Kongenitale myotone Dystrophie mit Zwerchfellparese", MONATSSCHRIFT KINDERHEILKUNDE ; ORGAN DER DEUTSCHEN GESELLSCHAFTFÜR KINDERHEILKUNDE UND JUGENDMEDIZIN ORGAN DER ÖSTERREICHISCHENGESELLSCHAFT FÜR KINDERUND JUGENDMEDIZIN, SPRINGER, BERLIN, DE, vol. 160, no. 12, 21 June 2012 (2012-06-21), pages 1236 - 1238, XP035150576, ISSN: 1433-0474, DOI: 10.1007/S00112-012-2721-6
  • [Y] SYR LEE ET AL: "Survival of a 30-week baby with congenital myotonic dystrophy initially ventilated for 55 days", JOURNAL OF PAEDIATRICS AND CHILD HEALTH, vol. 35, no. 3, 1 June 1999 (1999-06-01), pages 313 - 314, XP055186282, ISSN: 1034-4810, DOI: 10.1046/j.1440-1754.1999.00318.x
  • [Y] M A RUTHERFORD ET AL: "Congenital myotonic dystrophy: respiratory function at birth determines survival.", ARCHIVES OF DISEASE IN CHILDHOOD, vol. 64, no. 2, 1 February 1989 (1989-02-01), pages 191 - 195, XP055186288, ISSN: 0003-9888, DOI: 10.1136/adc.64.2.191
  • [A] GRUENER R ET AL: "Caffeine-modulated acetylcholine sensitivity in denervated rat and diseased human muscle", LIFE SCIENCES, PERGAMON PRESS, OXFORD, GB, vol. 17, no. 10, 15 November 1975 (1975-11-15), pages 1557 - 1565, XP025525591, ISSN: 0024-3205, [retrieved on 19751115], DOI: 10.1016/0024-3205(75)90177-0

Designated contracting state (EPC)

AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

Designated extension state (EPC)

BA ME

DOCDB simple family (publication)

EP 3020404 A1 20160518; CA 2966744 A1 20160519; EP 3217984 A1 20170920; EP 3217984 B1 20180912; ES 2722898 T3 20190819; US 2017304309 A1 20171026; WO 2016075288 A1 20160519

DOCDB simple family (application)

EP 14382450 A 20141114; CA 2966744 A 20151113; EP 15797633 A 20151113; EP 2015076553 W 20151113; ES 15797633 T 20151113; US 201515526207 A 20151113